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FDA's Patient-Focused Drug Development Initiative and the Benefit-Risk Assessment Framework


Evaluating the balance between the benefits and risks of new products under review is among the most important elements regulators juggle across the lifecycle of a product. And the question of how to bring more structure, rigor, consistency, and predictability to benefit-risk evaluation is receiving intense focus at the moment. In a FasterCures Webinar on the subject, moderator Cecilia Arradaza, FasterCures managing director of communications and policy, noted that there have been several meetings hosted by organizations such as the Institute of Medicine and the Drug Information Association on the topic just within the last month. “We’re moving toward a new paradigm here,” commented Kim McCleary, FasterCures’ director of strategic initiatives. “Everybody’s figuring out this landscape fresh.”

During the Webinar, Andrea Tan and Pujita Vaidya from the Office of Strategic Programs at the U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research, reviewed the status of two new efforts mandated by the 2013 Food and Drug Administration Safety and Innovation Act (FDASIA): a more structured framework for assessing the benefits and risks of products under review, and a new Patient-Focused Drug Development Initiative (PFDDI) that provides patients a formal pathway for providing input to regulators on the particulars of their conditions and current treatment options.

The two are intimately linked. The new Benefit-Risk Framework developed by FDA features five key decision factors that will inform reviewers’ decision-making:

  • Analysis of a condition;
  • Current treatment options;
  • Benefits of a treatment under review, as demonstrated in clinical trials;
  • Known risks of those products; and
  • Risk management.

FDA believes patients are uniquely positioned to inform the first two elements, which together create the “clinical context” for regulatory decision-making.

The Patient-Focused Drug Development Initiative creates a platform for FDA to gather this information from patients. The agency has selected 16 of the 20 disease areas on which to focus (and has hosted meetings on five of them to date), but Tan and Vaidya emphasized that the framework and process created and refined through PFDDI can and should be adopted by patient communities from other disease areas as well, and that they would be happy to discuss how such information might be supplied to them outside the context of the formal PFDDI meetings.

 “This has truly been an experimental process, “ noted Vaidya. FDA has tried to tailor each meeting to the specific disease community and has experimented with features meant to increase participation such as a “talk show-style” format, Webcasts, phone participation, and polling questions. The questions asked of patients are about symptoms and daily impacts that matter most to them and their perspectives on current treatment approaches – but again, questions are tailored to the specific diseases as hand. Effective planning and interaction with patient organizations have been critical to enhancing the success of these meetings.

After each PFDDI meeting, the agency prepares a “Voice of the Patient” report to capture the input of patients for consideration by FDA reviewers, but also to make this important information available to other stakeholders for whom it is valuable, including industry sponsors who might use it to help identify areas of unmet need or develop clinical outcomes tools.

Following FDA’s Webinar presentation, Eric Gascho, assistant vice president for government affairs at the National Health Council (NHC), informed participants about a new Patient Information Tool NHC has developed to help patient communities gather the kind of information important to both regulators and developers. The tool lays out a framework of questions and variables related to:

  1. Identification of subpopulations
  2. Description of disease diagnosis and impact (diagnosis, impact on subpopulations, impact on social factors, outcomes measures)
  3. Treatment and management impacts (availability, effectiveness, safety, use, access)

Gascho emphasized the need to better incorporate patient experience not only into regulatory decision-making but also into much earlier stages of development – the setting of research agendas, development of research questions, selection of outcomes and comparators, recruitment, and dissemination and translation. The tool can also be used by patient groups for internal purposes, to better understand patients’ needs and tailor programs to meet them. NHC is currently working on an implementation guide to help organizations maximize its use. 

McCleary praised FDA for having created “a safe place for patients to share their experiences” and noted that the PFDDI process was important not just for the information generated about 20 diseases but for “learning how to seek this input from patients.” She encouraged other patient groups to use the tools being developed by the FDA and the National Health Council to inform developers and regulators, citing work being done by Parent Project Muscular Dystrophy to pilot these methodologies in their own disease area. She called on patients to be prepared for these discussions and participate effectively in them. And she highlighted that it will be important in 2014 to watch how PFDDI and the new Benefit-Risk Framework are incorporated into the real-life review process.

Related FasterCures resources:

Related FDA resources: